Ontology highlight
ABSTRACT:
SUBMITTER: Poletto E
PROVIDER: S-EPMC7014411 | biostudies-literature | 2020 Jan
REPOSITORIES: biostudies-literature
Poletto Edina E Baldo Guilherme G Gomez-Ospina Natalia N
International journal of molecular sciences 20200113 2
Genome editing holds the promise of one-off and potentially curative therapies for many patients with genetic diseases. This is especially true for patients affected by mucopolysaccharidoses as the disease pathophysiology is amenable to correction using multiple approaches. Ex vivo and in vivo genome editing platforms have been tested primarily on MSPI and MPSII, with in vivo approaches having reached clinical testing in both diseases. Though we still await proof of efficacy in humans, the thera ...[more]